You might have seen news stories about gene silencing, and how the NHS has agreed to fund it as a treatment for a condition called amyloidosis. …
Simon Dadd new trustee
We are delighted to announce that Simon Dadd has joined our Board of Trustees. Simon has considerable knowledge of Duchenne, being the father of two …
Capricor publish interim results of HOPE-2 trial
Capricor therapeutics has announced the results of their interim analysis of the phase 2 trial of CAP-1002 in Duchenne. The trial has recruited 17 …
Rare disease genomes event
Last week Neil attended a “demystifying genomes for patient registries” event hosted by Sano genetics. The event was meant to give charities in the …
PM launches new drive to tackle barriers faced by disabled people
In June, the outgoing Prime Minister Theresa May announced a series of measures aimed at securing higher accessibility standards for new housing and …
PM launches new drive to tackle barriers faced by disabled peopleRead More
Conference registration open
Sign up today for the most amazing weekend of knowledge, power, support, education, community and hope. We've worked tirelessly to bring you the …
MARVEL headline sponsor for Superhero Series
The Superhero Series - the UK’s only mass-participation sports series dedicated to people with disabilities – will have a powerful boost this year, as …
Conference agenda released
We are excited to release our long awaited Agenda for #ADconf19! We have carefully aligned our Agenda this year with our three core objectives - …
Duchenne Dad cycles 282 miles in 24.5 hours
A big congratulations to Alexis Ellison who cycled an astonishing 282 miles in 24.5 hours this weekend, completely unsupported. 2 years ago Lex, …
EAMS renewed for Idebenone
Santhera Pharmaceuticals has announced that the UK’s medicines regulator (the MHRA) has renewed the Early Access to Medicines Scheme (EAMS) for …
CPG meeting in Scottish Parliament
Last week Shelley attended a Cross Party Group meeting at the Scottish Parliament with our Scottish Advocacy Officer, John Miller. They were joined at …
Stoke by Nayland charity golf day
On Saturday the 8th of June the Stoke by Nayland Golf Club men's captain, John Sandford, held his charity day in support of Action Duchenne. 34 …
